On September 7, 2016, Retrophin Inc. released the “Top Line” results from their recently completed DUET study, a Phase 2 clinical trial testing safety and efficacy of Sparsentan for FSGS patients.

Results showed promise for Sparsentan’s effectiveness at reducing proteinuria in patients with FSGS, with one group of patients seeing an average reduction of 44.8%. The DUET study included 96 patients, and only one serious adverse side effect (anemia) was reported. All patients chose to extend their treatment with Sparsentan during the trial’s open label extension period.

Alvin Shih MD, the executive vice-president for Retrophin Inc., said “significant reductions in proteinuria, along with a well-tolerated preliminary safety profile have us excited about being one step closer to providing a new treatment option for patients with FSGS.”

NephCure Kidney International is excited about these results and support Dr. Shih’s hope that we are moving closer to providing a new, effective, and safe treatment option for FSGS patients. Mark Stone, Chief Executive Officer of NephCure Kidney International, remarked “These preliminary results are very exciting for our community. This gives us hope that better treatment options will be available for our families in the near future.”

NephCure Kidney International would like to thank everyone who contributed time, talent, and resources to this study. Thank you, especially, to the patients and families who participated and helped bring effective treatments within reach.

The Obama Administration met with numerous companies, foundations, hospitals, universities, and patient advocacy groups at the White House’s Organ Summit on Monday, June 13. The goals of the summit include increasing the number of organ transplants by 2,000 each year, improving patient outcomes, facilitating research and developments around organ donation, and closing the gap between Americans who support organ donation and those who are actually registered organ donors.

Last year, the United States exceeded 30,000 annual organ transplants for the first time, yet 120,000 Americans are still waiting for an organ donation. Today, twenty-two people will die waiting for a life-saving transplant. President Obama, and several government and non-governmental organizations have made many efforts to reduce the organ donation waitlist, support patients, and increase access to organ transplantation.

Announced on Monday, almost $200 million in investments will be made to facilitate research and development related to organ donation. Specifically the Department of Defense (DOD) in a $160 million public-private investment will create an Advanced Tissue Biofabrication Manufacturing Innovation Institute to develop new manufacturing techniques to repair organ damage by replacing cells and tissues and that can hopefully be used one day to replace entire organs. In similar efforts, the DOD will award small businesses working to advance the science behind preserving organs and tissues.

The donor registration system is being re-imagined to seamlessly and effectively increase registrations and transplants. More than a dozen organizations including Facebook and Twitter are finding new tools and developing campaigns to make registering to be an organ donor easier with the intention of signing up 1 million new donors by fall of 2016.

More than 100,000 people on the organ waiting list are awaiting a kidney transplant so kidney-specific projects were a highlight of Monday’s Summit.

The American Society of Nephrology will partner with the XPRIZE Foundation to encourage the development of a new device solution for patients experiencing end-stage renal disease – an improvement on current dialysis methods. This project aims to overcome the decades of stagnation in kidney disease treatment.

In addition, dozens of transplant centers announced a collaboration to share data and best practices for hard-to-match patients, which could help more than 1,000 people gain access to transplants. Johns Hopkins University is currently working with the National Institute of Allergy and Infectious Disease to create HIV-positive donor pools, which could also lead to as many as 1,000 more transplants per year.

Part of the Organ Summit included the publication of letters written by organ donation recipients. One of which was from NBA player Alonzo Mourning, a kidney transplant patient. Mourning, who lost his kidney due to FSGS, went on to win a championship following his transplantation, and today brings awareness to the efforts of the White House and the importance of organ donation registration.

Mike and Kate Maurer are runners who came up with an inspired idea and organized a great event for a great cause, but they’re more than that: they’re parents to a wonderful little boy named Alex. Five-year-old Alex is active and energetic, he loves climbing and running…when he’s feeling well enough to play like other five year olds. Alex has Nephrotic Syndrome, a condition neither of his parents had even heard of before he was diagnosed.

Ultra-runner Troy Thompson

Mike and Kate know the trials of managing and living with NS, and like tens of thousands of others rely on breakthroughs in medical research to find better treatments and a cure for their son. They decided they needed to do something. As avid runners having completed numerous races of distances ranging from 5k to marathon, and team relays of 212 miles, they decided to blend their love of running with their desire to fundraise and created a 124-mile race called the Rival Game Relay. Capitalizing on a centuries-old Iowa rival between the University of Iowa Hawkeyes and the Iowa State Cyclones, they timed their race to coincide with the annual Iowa/ISU football game.

Eleven teams registered to complete the course beginning in Ames, IA, Cyclone territory, and finishing just outside of Iowa City, IA, land of the Hawkeyes. Several teams were comprised of eight or more runners who ran various legs, “ultra” teams contained 5 runners or less, and ultra runner Troy Thompson completed 102 of the 124 mile course on his own!

NephCure Kidney International thanks the Maurers and all of the runners who participated in the inaugural Rival Game Relay event.

NephCure Kidney International (NKI) rang the opening bell at the New York Stock Exchange (NYSE) at 9:30 AM Friday, August 15, 2014.

“We are thrilled to have been asked and for the opportunity to join the legions who have come before us in having this honor,” said Mark Stone, NephCure Kidney International CEO.

Representatives from NKI took the podium alongside NephCure Board Members and NephCure families including The Silvermans, The Genatt’s, Michael Levine, and Tim Brink. Five-year-old Jed Silverman had the honor of ringing the bell.

“This is an incredible opportunity for NephCure Kidney International. We appreciate the chance to raise awareness of the foundation, and its work to find a cure for the devastating kidney disease FSGS and the other diseases that cause Nephrotic Syndrome,” said Michael Levine, NKI Board Member. Mr. Levine’s 11-year-old son Matthew is currently battling FSGS (Focal Segmental Glomerulosclerosis).

More than 8,000 people are diagnosed with Nephrotic Syndrome every year, and FSGS is the leading cause of kidney failure in children. In the United States alone more than 26 million suffer from some form of kidney disease. Since its inception, NephCure has contributed more than $13 million dollars toward FSGS and NS research. NKI remains the only organization dedicated to funding research to end FSGS and NS.

Organizational Mission

NephCure Kidney International ® is the only organization committed exclusively to support research seeking the cause of the potentially debilitating kidney disease Focal Segmental Glomerulosclerosis (FSGS) and Nephrotic Syndrome, improve treatment and find a cure.